A Randomized Study of Autologous Umbilical Cord Blood Reinfusion in Children With Cerebral Palsy

Joanne Kurtzberg, MD

Status and phase: Completed; Phase 2

Conditions: Spastic Cerebral Palsy; Cerebral Palsy; CP

Treatments: Other: Placebo; Biological: Autologous UCB Reinfusion

Study type: Interventional

Funder types: Other

Identifiers

NCT01147653

Pro00017801

Details and patient eligibility

About

The purpose of this study is to determine the efficacy of a single intravenous infusion of autologous umbilical cord blood (UCB) for the treatment of pediatric patients with spastic cerebral palsy.

Full description

Cerebral palsy results from in utero or perinatal injury to the developing brain, often through stroke, hypoxic insult or hemorrhage. Currently available treatments for patients with cerebral palsy are supportive, but not curative. Umbilical cord blood (UCB) has been shown to lessen the clinical and radiographic impact of hypoxic brain injury and stroke in animal models. UCB also engrafts and differentiates in brain, facilitating neural cell repair, in animal models and human patients with inborn errors of metabolism undergoing allogeneic, unrelated donor UCB transplantation. We hypothesize that, in the setting of brain injury, infusion of autologous UCB will facilitate neural cell repair resulting in improved function in pediatric patients with cerebral palsy.

Enrollment: 63 patients

Sex: All

Ages: 12 months to 6 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Age ≥ 12 months and ≤ 6 years
• Diagnosis: Spastic cerebral palsy with diplegia, hemiplegia, or quadraplegia.
• Performance status:

Gross Motor Function (GMF) Classification Score levels II - IV or GMF Score leve I, age >/= 2 years Spastic hemiplegia: GMF Score II-IV or minimal functional capabilities in the affected upper extremity. A subject classified as GMFCS level I with significant upper extremity impairment will be eligible if the affected upper extremity is used as an assist only. An eligibility committee will meet to review the child's records and determine eligibility.

Bilateral hypotonic CP (diplegia or quadriplegia): GMF Score II-IV and an abnormal brain MRI suggestive of an acquired etiology (versus a genetic etiology or brain malformation).

• Autologous umbilical cord blood available at a private or public cord blood bank with a minimum total nucleated cell dose of ≥ 1 x 107 cells/kilogram.
• Parental consent.

Exclusion criteria

• Athetoid cerebral palsy.

• Autism and autistic spectrum disorders without motor disability.

• Hypsarrhythmia.

• Intractable seizures causing epileptic encephalopathy.

• Evidence of a progressive neurologic disease.

• Known HIV or uncontrolled bacterial, fungal, or viral infections.

• Impaired renal or liver function as determined by serum creatinine >1.5mg/dL and/or total bilirubin >1.3mg/dL.

• Head circumference >3 standard deviations below the mean for age.

• Known genetic disease or phenotypic evidence of a genetic disease on physical examination.

• Concurrent genetic or acquired disease or comorbidity(ies) that could require a future allogeneic stem cell transplant.

• Requires ventilatory support, including home ventilator, CPAP, BiPAP, or supplemental oxygen.

• Patient's medical condition does not permit safe travel.

• Previously received any form of cellular therapy.

• Autologous umbilical cord blood unit has any of the following:

  1. Total nuclear cell dose < 1 x 107 cells/kilogram
  2. Positive maternal infectious disease markers (except CMV)
  3. Evidence of infectious contamination of the cord blood unit
  4. Lack of a test sample to confirm identity
  5. Evidence of a genetic disease

• Unable to obtain parental consent.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Quadruple Blind

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63 participants in 2 patient groups, including a placebo group

Autologous UCB Reinfusion First,Then Placebo

Active Comparator group

Description:

Subjects receive their autologous umbilical cord blood cells at Baseline, than placebo at Year 1.

Treatment:

Other: Placebo

Biological: Autologous UCB Reinfusion

Placebo First, Then Autologous UCB Reinfusion

Placebo Comparator group

Description:

Subjects receive placebo at Baseline, then autologous umbilical cord blood cell reinfusion at Year 1.

Treatment:

Other: Placebo

Biological: Autologous UCB Reinfusion